Current Research


What we are funding now

Our ultimate goal is to live in a world without Glycogen Storage Disease. 

Your generous contributions are working hard to help us get there.


Our funding supports innovative research that results in groundbreaking discoveries and hope for the future.

Gene Therapy

On July 24, 2018, we reached a milestone when the first dose of gene therapy was delivered to a GSD patient. The GSD team at the University of Connecticut is leading the study and initial results are extremely positive.  Phase 3 of this trial will commence in January 2022.

Gene Editing

CRISPR/Cas9- based gene editing system allows researchers to edit DNA sequences and modify gene function. Many scientists believe it is a novel approach to curing genetic disorders. The Children’s Fund for GSD Research is currently funding a study led by Dr. Janice Chou at The National Institute of Health (NIH) to determine if this is a safe and viable option to cure Glycogen Storage Disease.

Prevention of Adenomas

Hepatocellular adenoma/carcinoma (HCA/HCC) is a long-term complication of GSDIa. Scientists at The University of Connecticut are conducting studies to determine why and the best way to prevent this.


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" ... because every child deserves to be healthy."

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